Syndicate content

“Exceptional Results” Suggest That CAR T Cell Therapy Could Become New Standard of Care for Patients with Refractory Aggressive Lymphoma

On February 28, 2017, Kite Pharma, Inc., (Nasdaq:KITE) announced positive data from the primary analysis of ZUMA-1 for its lead CAR-T candidate, axicabtagene ciloleucel (previously referred to as KTE-C19), in patients with chemorefractory aggressive B-cell non-Hodgkin lymphoma (NHL). The study of 101 patients met the primary endpoint of objective response rate (ORR), or rates of tumor response (complete response + partial response) recorded after a single infusion of axicabtagene ciloleucel, with 82 percent (p < 0.0001). 41% of patients were in response and 36% were in complete response (CR) at month 6 of the study. According to Kite, these results demonstrate the treatment effect of axicabtagene ciloleucel in a patient population with multiple types of aggressive NHL, including diffuse large B-cell lymphoma (DLBCL) enrolled in Cohort 1, as well as primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL) enrolled in Cohort 2. "These results with axicabtagene ciloleucel are exceptional and suggest that more than a third of patients with refractory aggressive NHL could potentially be cured after a single infusion of axicabtagene ciloleucel," said Jeff Wiezorek, M.D., Senior Vice President of Clinical Development. "The ZUMA-1 study was built on a foundation of support and commitment from Dr. Steven Rosenberg and the National Cancer Institute and our ZUMA-1 clinical trial investigators who believed in the potential for CAR-T therapy to change the paradigm of cancer treatment." “This seems extraordinary ... extremely encouraging,” said one independent expert, Dr. Roy Herbst, cancer medicines chief at the Yale Cancer Center, as quoted in a Washington Post article (see link below) on the Kite announcement. The Post article explained that “the treatment involves filtering a patient’s blood to remove key immune system soldiers called T-cells, altering them in the lab to contain a gene that targets cancer, and giving them back intravenously. Doctors call it a ‘living drug’ — permanently altered cells that multiply in the body into an army to fight the disease.”

Kite hosted a live conference call and webcast on February 28, 2017. The webcast will be made available on the Company's website at www.kitepharma.com under the Investors tab in the Events and Presentations section. A replay of the live audio webcast will be available on the Company's website for approximately 30 days.

AXICABTAGENE CILOLEUCEL

Kite Pharma's lead product candidate, axicabtagene ciloleucel, is an investigational therapy in which a patient's T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells.

Axicabtagene ciloleucel has been granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.

Kite intends to seek regulatory approval of axicabtagene ciloleucel in aggressive NHL based upon the combined data from all 101 patients and plans to complete its rolling submission of the Biologics License Application (BLA) by the end of the first quarter of 2017. In addition, Kite plans to submit a marketing authorization application (MAA) for axicabtagene ciloleucel for the treatment of relapsed or refractory DLBCL, PMBCL and TFL with the European Medicines Agency (EMA) in 2017.

POSSIBLE NEW STANDARD OF CARE

"We know as clinicians that patients with aggressive lymphoma who do not respond to their previous treatments have a very poor prognosis. In fact, we know from the SCHOLAR-1 study, these patients have only an eight percent chance of achieving a complete response with current therapies," said Frederick L. Locke, M.D., ZUMA-1 Co-Lead Investigator, and Director of Research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, Florida. "Several patients we treated at Moffitt Cancer Center experienced a rapid and durable complete response with this first-of-its kind therapy. The ZUMA-1 study results suggest that axicabtagene ciloleucel could become a new standard of care for patients with refractory aggressive lymphoma."

Sattva S. Neelapu, M.D., Department of Lymphoma/Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, served as a co-lead investigator in the ZUMA-1 trial.

Full data from the primary analysis will be presented at the American Association for Cancer Research (AACR) in April 2017 in Washington, D.C.

ZUMA-1 is supported in part by funding from The Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program®.

TWO DEATHS DEEMED RELATED TO AXICABTAGENE CILOLEUCEL

As previously reported at the American Society of Hematology Annual Meeting in 2016, there were three deaths not due to disease progression in the study. Two events, one hemophagocytic lymphohistiocytosis and one cardiac arrest in the setting of cytokine release syndrome (CRS), were deemed related to axicabtagene ciloleucel. The third case, a pulmonary embolism, was deemed unrelated. Between the interim analysis that included 62 patients, and this primary analysis which now includes all 101 patients, there were no additional deaths due to adverse events.

KITE PHARMA

Kite is a biopharmaceutical company engaged in the development of innovative cancer immunotherapies with a goal of providing rapid, long-term durable response and eliminating the burden of chronic care. The company is focused on chimeric antigen receptor (CAR) and T cell receptor (TCR) engineered cell therapies designed to empower the immune system's ability to recognize and kill tumors. Kite is based in Santa Monica, CA. For more information on Kite, please visit www.kitepharma.com. Sign up to follow @KitePharma on Twitter at www.twitter.com/kitepharma.

[Press release] [Reuters article] [Washinvgton Post article] [The Telegraph article] [Seattle Times article] [CBS News article] [Genetic Engineering & Biotechnology News article] [The Street.com article]